Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)

PepGen

Status and phase: Enrolling; Phase 1

Conditions: Myotonic Dystrophy 1

Treatments: Other: Placebo; Drug: PGN-EDODM1

Study type: Interventional

Funder types: Industry

Identifiers

NCT06204809

PGN-EDODM1-101

Details and patient eligibility

About

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).

The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

Enrollment: 24 estimated patients

Sex: All

Ages: 18 to 50 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
• Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
• Presence of myotonia

Exclusion criteria

• Congenital DM1
• Known history or presence of any clinically significant conditions that may interfere with study safety assessments
• Abnormal laboratory tests at screening
• Medications specific for the treatment of myotonia within 2 weeks prior to screening
• Percent predicted forced vital capacity (FVC) <40%

Note: Other inclusion and exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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24 participants in 4 patient groups, including a placebo group

Single Dose of PGN-EDODM1 at Dose Level 1

Experimental group

Treatment:

Drug: PGN-EDODM1

Single Dose of PGN-EDODM1 at Dose Level 2

Experimental group

Treatment:

Drug: PGN-EDODM1

Single Dose of PGN-EDODM1 at Dose Level 3

Experimental group

Treatment:

Drug: PGN-EDODM1

Single Dose of Placebo

Placebo Comparator group

Treatment:

Other: Placebo